A Study looking at the long-term use of a CFTR modulator (Ivacaftor) in children with cystic fibrosis who are less than 24 months and have a CFTR gating mutation


Therapeutic category
Restore CFTR Function
Trial status
closed Participating Centres
Phase III

Full title

A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation
Trial Reference Number
Trial type
Length of participation
104 weeks
CFTR Modulator (Ivacaftor)
Last edited date
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type

Who can take part?

Top inclusion criteria
  • Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
  • An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older
Top exclusion criteria
  • Subjects receiving commercially available ivacaftor treatment

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